The cholic acid extension study in Zellweger spectrum disorders: results and implications for therapy

ConclusionsAlthough CA treatment did lead to reduced levels of toxic C27-bile acid intermediates in ZSD patients without severe liver fibrosis or cirrhosis, no improvement of clinically relevant parameters was observed after 21  months of treatment. We discuss the implications for CA therapy in ZSD based on these results.
Source: Journal of Inherited Metabolic Disease - Category: Internal Medicine Source Type: research